Urea Cycle Disorders – Drugs In Development, 2024
Powered by ![]()
Unlock hidden opportunities in the Pharmaceuticals industry
Empower your strategies with our Urea Cycle Disorders – Drugs In Development, 2024 report and make more profitable business decisions.
Urea cycle disorders (UCDs) are a group of inherited metabolic diseases that affect the ability to remove excess nitrogen from the body. Nitrogen is a by-product of protein breakdown and is normally converted into urea, a harmless substance that is excreted in urine. However, in UCDs, one of the enzymes or transporters involved in the urea cycle is defective, leading to the accumulation of ammonia, a toxic substance, in the blood and brain. This can cause serious neurological problems, such as seizures, coma, and brain damage. UCDs are rare, affecting about 1 in 35,000 newborns. There are eight types of UCDs, each named after the deficient enzyme or transporter. They are N-acetylglutamate synthase (NAGS) deficiency, carbamoyl phosphate synthetase I (CPS1) deficiency, ornithine transcarbamylase (OTC) deficiency, argininosuccinate synthase 1 (ASS1) deficiency or Citrullinemia type I, citrin deficiency or citrullinemia type II, argininosuccinic lyase (ASL) deficiency, arginase (ARG) deficiency, and ornithine translocase deficiency.
The Urea Cycle Disorders drugs in development market research report provide comprehensive information on the therapeutics under development for Urea Cycle Disorders, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. The report also covers the descriptive pharmacological action and product description of the therapeutics, and the latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Urea Cycle Disorders and features dormant and discontinued products.
Note:
* This is an “on-demand” report and will be delivered within 2 business days (excluding weekends and holidays) of the purchase.
* Certain sections in the report may be removed or altered based on the availability and relevance of data.
| Quick View – Urea Cycle Disorders | Key Targets |
|
|
| Key Mechanisms of Action |
|
||
| Key Routes of Administration |
|
||
| Key Molecule Types |
|
||
| Major Companies |
|
||
Scope
- Therapeutics in Development: Covering 36 molecules, with 34 developed by companies and the rest by universities/institutes.
- Analysis Parameters: Offering insights by stage of development, drug target, MoA, RoA, and molecule type for a comprehensive overview.
- Pharmacological Insights: Understand the descriptive pharmacological action of therapeutics.
- Development History: Access the complete research and development history.
- Latest News and Press Releases: Stay updated with the latest developments through comprehensive news coverage.
Reasons to Buy
- Holistic insights: Understand the broad spectrum of Urea Cycle Disorders therapeutics, aiding strategic decision-making with insights into stages, targets, MoA, RoA and molecule types.
- Pipeline Exploration: Explore the detailed Urea Cycle Disorders pipeline, offering nuanced analysis of drug targets, mechanisms of action and routes of administration for varied decision-making.
- Comprehensive R&D: Access thorough R&D histories, providing a comprehensive understanding of Urea Cycle Disorders treatments for adaptable decision-making.
- Save valuable hours: Identify key players steering innovation in therapeutics, enabling strategic partnerships.
Key Players
Arcturus Therapeutics Holdings IncAvanti Biosciences Inc
Bio-Cancer Treatment International Ltd
Bloomsbury Genetic Therapies Ltd
Boehringer Ingelheim International GmbH
Callitas Therapeutics Inc
CAMP4 Therapeutics Corp
Carbiotix AB
Cellaion SA
Cycle Pharmaceuticals Ltd
Evox Therapeutics Ltd
Gene Therapy Research Institution Co Ltd
Genfit SA
HemoShear Therapeutics LLC
iECURE
Insmed Inc
Ironwood Pharmaceuticals Inc
Liberyx Therapeutics Ltd
Moderna Inc
NanoCell Therapeutics Inc
OrphanPacific Inc
Penn Medicine
PHAXIAM Therapeutics SA
Poseida Therapeutics Inc
Relief Therapeutics Holding AG
Sanofi
Scarlet Therapeutics Ltd
Spyre Therapeutics Inc
TenNor Therapeutics Ltd
UCLA Technology Development Group
Ultragenyx Pharmaceutical Inc
Unicyte AG
University of California Los Angeles
Vivet Therapeutics SAS
Table of Contents
Table
Figures
Frequently asked questions
Related reports
View more Urea Cycle Disorders reports